Children’s National Hospital Pioneers Access to Life-Changing Gene Therapies

In a breakthrough that’s transforming lives at Children’s National Hospital in Washington, D.C., gene therapy emerges as a beacon of hope. Eric Manuel Balmir, PharmD, the hospital’s Vice President of Clinical Ancillary Services and Chief Pharmacy Officer, shares compelling narratives of its impact. Gene therapy, a revolutionary approach, involves editing the patient’s genome to replace defective DNA strands. This innovative treatment is currently addressing a range of diseases, including spinal muscular atrophy, a condition with muscular dystrophy-like symptoms.

Balmir recounts an inspiring story: a young patient, once wheelchair-bound due to spinal muscular atrophy, walked out of the hospital post-treatment. “Pharmacy has evolved,” Balmir notes. “We’ve moved from merely managing symptoms to targeting the root cause of diseases.” Highlighting the evolution, he points out the significant advancements in treating conditions like hypertension and diabetes, which are now saving more lives than ever.

However, the path to gene therapy is not without its challenges, primarily due to the staggering costs involved. The treatment for spinal muscular atrophy, for example, is onasemnogene abeparvovec-xioi, also known as Zolgensma, which comes with a hefty price tag of $2.1 million per patient. Despite the high costs, the promise of gene therapy is undeniable, with recent FDA approvals like Casgevy and Lyfgenia marking significant milestones for sickle cell disease treatment in patients aged 12 and older.

The FDA’s pipeline anticipates 54 gene and cell therapy approvals by 2030, underscoring the urgent need for a sustainable coverage pathway. Balmir emphasizes the importance of making these life-saving treatments accessible, noting that other countries have already taken steps in this direction. Children’s National is actively engaging with legislators to advocate for coverage of these expensive therapies.

To address accessibility challenges, Children’s National has partnered with Clearway Health, a specialty pharmacy solutions provider. Clearway Health serves as a crucial intermediary, securing payer approvals and facilitating access to gene therapy drugs. This partnership was instrumental when the hospital administered ZYNTEGLO, a gene therapy for transfusion-dependent beta-thalassemia, showcasing a model that balances the high costs while maintaining the hospital’s financial health.

Clearway Health’s innovative approach not only streamlines the procurement process for novel gene therapies but also enhances the hospital’s capability to offer cutting-edge treatments. “Many health systems struggle with the complexities of acquiring a new gene therapy,” Balmir states. “Clearway Health’s framework enables us to navigate these challenges efficiently, ensuring our patients have access to the latest pharmaceutical advancements.”

Furthermore, Clearway Health’s commitment to building in-house specialty pharmacies within hospitals marks a significant step towards integrated, patient-centered care. By fostering stronger ties with manufacturers, payers, and pharmacy benefit managers, Clearway Health aims to negotiate better costs, secure limited distribution medications, and improve access to essential treatments.

As Shawn Francis, PharmD, Director of Specialty Pharmacy at Clearway Health, points out, novel gene therapies hold the promise of liberating patients from lifelong, costly care with a single treatment. However, questions about coverage, cost-effectiveness, and delivery remain. The journey of gene therapy, from a groundbreaking concept to a mainstream treatment, symbolizes a pivotal shift in medical science, offering hope and new beginnings to many.

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